Clinical Observation of Luspatercept in Treatment of Chinese Adult β-thalassaemia Patients With TD β-thalassemia

NCT06164821 | Unknown

• 1 other identifier: B-thal01
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

To assesse the efficacy and safety of luspatercept versus placebo in China patients with transfusion-dependent β-thalassaemia.

Conditions

Effect of Drug

Keywords

luspatercept; β-Thalassemia

Outcome Measures

Primary Outcomes (2)

• Number of units in cumulative transfusion volume

  The decrease of cumulative blood transfusion volume in low transfusion burden group (\<7.5 units /24 weeks) and high transfusion burden group (7.5-15 units /24 weeks) after 24 weeks of treatment;

  Within 24 weeks

• Proportion of patients with a 33% reduction in transfusion burden

  33% reduction in blood transfusion burden at week 24 in low transfusion burden group (\<7.5 units /24 weeks) and high transfusion burden group (7.5-15 units /24 weeks);

  Within 24 weeks

Secondary Outcomes (2)

• Blood transfusion burden reduction ratio

  Within 24 weeks

• Ratio of TI

  Within 24 weeks

Other Outcomes (1)

• Security Index

  Within 33 weeks

Study Arms (1)

luspatercept arm

EXPERIMENTAL

Luspatercept was given once subcutaneously every 3 weeks for 24 weeks in the treatment period, . Luspatercept was started at 1·0 mg/kg with titration up to 1·25 mg/kg, or reduction in the event of toxicity or excessive haemoglobin concentration increase.. During the treatment, the hemoglobin of patients before each injection of luspatercept should be monitored, and the common adverse reactions (AE) should be monitored. According to the judgment and practice of clinicians, the best supportive treatment, including blood transfusion, iron chelation therapy, and anti-infection treatment, should be provided for patients receiving luspatercept treatment. If the patient has blood transfusion, it is necessary to obtain the blood transfusion record from the hospital system.Concomitant use of iron chelating agents was also recorded.

Drug: luspatercept

Interventions

luspatercept DRUG

Adult patients with TD thalassemia were given luspatercept subcutaneous injection.

luspatercept arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥18 years old;
• A clear diagnosis of transfusion-dependent β-thalassemia (including αβ mixed type) with red blood cell transfusion ≤15u within 24 weeks before enrollment (one unit of red blood cell in overseas clinical research is 200-350ml packed red blood cells, which should be converted according to Chinese clinical practice);
• Voluntarily participate in the study and sign the informed consent;

You may not qualify if:

• pregnant or lactating women;
• Allergic to luspatercept and/or luspatercept for injection excipients;
• Severe liver dysfunction: Liver enzymes (alanine aminotransferase ALT or aspartate aminotransferase AST) ≥ 3 times normal value.;
• Severe renal injury: eGFR\<30 ml/min/1.73m3 or end-stage renal disease;
• heart disease, New York Heart Association (NYHA) class 3 or higher heart failure, or the need for treatment Severe arrhythmia, or recent myocardial infarction within 6 months;
• The patient had uncontrolled hypertension;
• Patients with a history of deep vein thrombosis or stroke within 24 weeks prior to enrollment.;
• Treatment with ESA, luspatercept, thalidomide or hydroxyurea within 12 weeks before enrollment;
• Any significant other medical condition, laboratory abnormality, or mental illness;
• Investigators deemed enrollment inappropriate.

Sponsors & Collaborators

• Hematology department of the 920th hospital: lead

Study Sites (1)

920th Hospital of Joint Logistics Support Force of People's Liberation Army of China

Kunming, Yunnan, 650000, China

RECRUITING

MeSH Terms

Interventions

luspatercept

Central Study Contacts

Chaoen Zhen, MD

CONTACT

18087991755; zce163163@163.com

Liu Liu, MD

CONTACT

0871-64774206

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: 5 cases with transfusion burden\<7.5 units/24 weeks, and 5 cases with transfusion burden 7.5-15 units/24 weeks

Study Record Dates

• First Submitted: December 1, 2023
• First Posted: December 11, 2023
• Study Start: January 1, 2024
• Primary Completion: January 1, 2024
• Study Completion: November 30, 2024
• Last Updated: December 22, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will share

Locations

CN (1)